Enterprises of Great Pitch and Moment

Before all this happened, I wrote plays. Before Susannah was diagnosed with a rare neurodegenerative disease; before we started the foundation; before I knew about motor proteins and phenotypes; before special needs; before rare disease policy; before biotech; before that moment in the hospital elevator when everything changed, one of my jobs was teaching high school and college-level playwrighting.

Over the course of a semester we covered what I believe to be the 12 elements needed to write a good play. The first day of class focused on four key elements of playwriting: two questions and two directives. If a play is crafted around these four principals, it will be a strong story that impacts the audience and encourages bold thought and action.

I have not written a play or walked onto a television or film set since just after our daughter’s diagnosis. I did return to the stage once to perform Love Letters as a benefit for the TREATMENT Center for Rare Pediatric Genetic Diseases. Last week as I sat in a midtown boardroom about to pitch one of three KIF1A research strategies and ask a company for 2.5 million dollars to help cure my kid, it struck me: the four key elements of playwriting I once preached to aspiring students on the first day of class could help me today. Perhaps if I take the structure used to craft a powerful play, people in the position to make KIF1A research and development possible will understand the importance of their role. Maybe the people I am about to ask to fund this urgent and innovative clinical program (a program designed by the most capable scientists and physicians to develop treatment for Susannah and kids like her) will dig deep, be bold and join our mission before it’s too late. (Pause) Two minutes before three people kind enough to take this meeting entered the room, I decided to test my hypothesis and thought hard to recall the last time I was in a classroom. Just over three years ago. Before all this happened.

I’ll let you in on a little secret,
a devastating fault I have – a weakness that prevents me from quickly funding critical
research aimed at curing our daughter and kids like her – for all the noise I
make as a rare disease advocate, I lack the most important skillset needed to
run a rare disease foundation: fundraising. I fail at nonprofit 101. I get
angry when people say no to our foundation because KIF1A Associated
Neurological Disorder is “too rare.” I am devastated when funds are not
allocated to research that will bring treatment to kids suffering from a
disease with no therapeutic options. I am baffled when philanthropists pass on
donating to our robust clinical research program; a program that could prevent
catastrophic seizures in the middle of the night and stop parents from walking
into their child’s room in the morning only to find a lifeless body.

The structure of rare disease fundraising
lacks empathy and urgency, and the playing field is not level. Industrial,
medical and scientific organizations with resources need to ask an important
question: why does the burden to fund early research and even development fall
on rare disease families? Why do parents of severely sick children have to sell
their cars, pull kids from private school, mortgage houses, deplete saving
accounts and (most difficult) spend time away from our children with complex
medical needs to go on a round of fundraising – precious time away from
children who rely completely on our care? One shouldn’t need to be convinced
that diseases without any treatment options need to be prioritized and funded, yet
every rare disease foundation and so many families spend their days anxiously
working to convince people to help before the toll of disease is beyond repair.
Perhaps it’s because these very people I pitch to don’t understand the
immediate impact their financial support will have. Too often those capable of
saying yes need to be convinced when I ask for help. Because time is as
much the enemy as nature, my iron fist lacks a velvet glove and my ask is never
as nuanced as it should be. I don’t have time for tact, we need funds to access
and apply existing science to KIF1A and save the lives of children living with
rare disorders; children like Susannah.

As I sat in that boardroom ready to
ask for funds needed to hire a specific team to design a viral vector – a
precision approach to deliver a healthy KIF1A gene (KIF1A is a problematically large
gene) to our children’s failing central nervous system, it hit me: maybe I
should apply those four key elements of playwriting from the first day of class
to this “ask” for KIF1A funding. Two questions and two directives critical in
crafting a powerful story and inspiring action. (Pause) So, I did.

Of course, the stakes of our
scientific and medical mission are exponentially higher than Hamlet’s plight
against Claudius or the lost dreams and dead seagulls in a Chekhov play. As
long as I don’t speak to these titans of industry in iambic pentameter, maybe the
molecular biology will make sense. Maybe these potential donors will realize
their return on investment is far greater than a downstream financial gain –
maybe these people will understand the return on investment is seeing Susannah
and kids like her walk.

The key elements of playwriting I
asked students to consider on the first day of class are:

What
do characters in your scene want and need from each other?

What
are your character’s secrets?

Write
an emergency: force characters to act with urgency.

Write
non-verbal dialogue.

Two questions and two directives. If
a playwright can answer those two questions and hit those two directives, he or
she will inspire action with their words. (Pause) Worth a shot.

Another fault of mine: I far prefer
tactics over strategy, and wear everything on my sleeve. I usually end up with
tears in my eyes and it’s certainly not by design. I’m not an entrepreneur and
I only care about one thing: finding treatment for Susannah and kids like her
with rare genetic diseases. So when three executives walked in, I told them
about my former life as a playwright and employed a playwriting 101 lesson plan
to accomplish what I went there to do: secure funds for the design of a viral
vector, and to show them a thorough and robust proof of concept to support my
ask.

I wrote the four key elements of playwriting on a whiteboard and addressed each. The first two are questions. I answered each with complete candor, articulating very personal moments for the first time. Then I reinforced our urgent need to fund this project – a project crafted by the most capable, innovative and passionate scientists in the world. It went like this:

1. What do characters in your scene want and need from each other?

I want you to understand that you are in a position to save the lives of children who have no treatment options and who are losing the battle with a degenerative disease. You will be part of our community and the decision you make today will immediately impact patients and families.

I need you to understand that the science to help children like Susannah already exists. There are two things standing in our way: a lack of money and a lack of time. You can solve both, and KIF1A is the tip of an iceberg that will lead to treatment for thousands of people with rare diseases.

2. What are your character’s secrets? (Pause)

I can’t remember the last time my wife and I slept in the same bed together. One of us sleeps next to Susannah every night because we’re afraid she might have a seizure in her sleep and die.

When we started the foundation, I promised myself that every time tragedy strikes and a child with KIF1A dies I will go to his or her funeral and hug the parents. In the last three years I have attended too many funerals of KIF1A children. (Pause) But I didn’t keep my promise to be at every funeral. We became very close with the family of a child in Sweden who had KIF1A, her name was Eleonora. When the family visited New York last year, my wife held Eleonora on her lap and sang “Wheels on the Bus” so softly. Eleonora smiled and her father told us it was very rare to see Eleonora respond with joy. I fell in love with this sweet girl. When Eleonora died in February, part of me died. I bought a plane ticket to Sweden to attend Eleonora’s funeral and had every intention of keeping that promise I made. When it was time to leave, I couldn’t stop crying and remembered my wife singing “Wheels on the Bus” and imagined Susannah playing with Eleonora. It hurts so much to think of Eleonora’s death and the emptiness her family must feel, an emptiness every mom and dad who lost a child must feel. (Pause) I didn’t go to the airport. I didn’t go to Eleonora’s funeral. I didn’t keep my promise. That’s a secret and something I will never forgive myself for.

3. Write an emergency: force characters to act with urgency.

This emergency was written for us. Susannah’s KIF1A gene has a complex, gain-of-function mutation that causes her brain and body to atrophy. Biotech and pharma have capabilities to develop therapies; the science exists. Pipelines are crowded, and our growing population is still too small to generate real interest from industry. Companies won’t prioritize a treatment for Susannah and KIF1A, so we are urgently raising money to do it ourselves. This is an emergency.

4.Write non-verbal dialogue.

In playwriting, non-verbal dialogue is magic. It’s that quiet moment when people on stage truly connect. This happens when characters listen and react to each other – when a playwright creates present and human characters capable of love. The playwright accomplishes this by writing in the word pause or silence in parenthesis between lines. That is non-verbal dialogue in theater. (Pause) To rare disease families non-verbal dialogue means something entirely different. Let me tell you a quick story:

Like many kids with KIF1A, Susannah has trouble communicating, especially in the afternoon. As the disease progresses, KIF1A kids have more seizures and extreme fatigue becomes a constant challenge. By mid-day she is wiped out and our noisy little girl sits in silence, too sore to walk and too tired to use the words she knows. Susannah has an epic amount of feelings in her soul and words stuck at the tip of her tongue. She works relentlessly to power each past a foggy stutter. Our superhero wants desperately to run, talk, laugh and play with the other kids.

Last week we were in Cape Cod with family and friends. The yard was teeming with cousins and neighbors. Susannah sat in her chair tired and sore, silent and having absent seizures. I sat beside her rambling about baseball and making silly faces – anything to keep her engaged and present. In front of us five carefree children (some younger than Sus) skipped in the sprinklers shouting and laughing, savoring childhood in summertime. It’s excruciating to see our daughter watch other children play and laugh. Susannah looked at me and I knew how desperately she wanted to scream out, laugh, jump out of her chair and sprint into the water with her cousins and friends. But she can’t. KIF1A keeps her too tired, spastic and silent. I looked back at Susannah without saying a word and knew for a fleeting moment she saw how scared I was; how scared I always am. She burst into tears, unable to verbalize pain and frustration. It’s crushing to see fear and confusion in Susannah’s face because she knows what is happening to her own body. Susannah doesn’t know the bliss and freedom that every child is entitled to, the inalienable human right to be a carefree kid on Cape Cod in July. Those carefree childhood summers don’t exist for kids like Susannah. Yet.

For KIF1A families, and so many others living with rare
genetic diseases, that is what non-verbal dialogue truly is. A helpless
interpretation of the pain our kids feel and the overwhelming emotions inside
of them. Feelings trapped inside quite literally dying to come out. (Pause)
All the while we do everything possible to raise funds needed to urgently execute
this well constructed, scientifically sound plan to bring treatment to Susannah
and children like her. The science exists. We need your help. We need 2.5
million dollars, and that’s just to get the work started – so I’ll be back
after we finish the first act of this play, together.

I thanked my hosts for their time and handed them a folder of relevant data and publications. On my way out the door I remembered the picture in my wallet – a picture I carry of Susannah and Eleonora taken the day our families met. A moment captured when I glanced across the room and saw Sally singing “wheels on the Bus” to our girls. The moment Eleonora smiled. (Pause) I turned to the people in that boardroom and just as I was about to show them the picture, I stopped. Instead I just smiled and walked out of the room. That moment is all mine for now. (Pause) I thought of Eleonora, Evie, Ryan, Brayden, Gustav and all the kids we’ve lost in the last three years – and I raced home to hug Susannah with all my might.

Comments

I couldn’t be more proud of how my friends and fellow artists are using your talents for the most important thing in the world. Luke and Sally have always risen above the rest; now you are flying. Keep singing, keep writing, keep storytelling, keep feeling, keep raising your kids to be just like you.

I too followed Eleonora and cried when she died. My little great granddaughter is now 8 and we pray people will help so these children can have a life. Your words are powerful. I thank you for all you do.

There are so few people who understand the sleepless nights , the heartache watching other children play completing basic skills that people take for granted. The sheer sadness when yet another child loses the fight. I wish we were coming to New York, just to say thankyou. Thankyou for all your efforts Luke.

Beautiful. Outstanding. Heartwrenching. Brilliant.
I read somewhere that intelligence is basically making connections between informations from different spheres, we have an obvious example here with you using your skills from your past life… to help save lives of our KIF1A children — and so many others who will eventually benefit from the therapy we are trying to test.
Lhassa won’t live long enough to benefit from your work. But I LOVE YOU for doing it anyway. Sending you all my admiration, respect, strength ans solidarity.

I am Lhassa’s grand-mother and, like my daughter Anouk, I was deeply moved by reading you. Your dauhgter is a beauty. Lhassa is one of the most beautiful baby I ever saw. During my daughter’s pregnancy, all the tests were indicating the Anouk would have a “top shape” baby. All the tests at her bearth said the same. We were all in heaven. I was the grand-mother of 3 when Lhassa was born but it took me 5 monthes to realised that something was wrong in her development…First I thought that she was blind…it would have been a blessing. When I think of my daughter, my son in law and their son Éli who loves so much his little sister, my heart is falling apart. Lhassa is not as conscient as Suzannah is but seing her suffering when she has problem to breath, fever is heartbreaking. THANK YOU so much to fight for children like yours and ours. Thank you for those who will benefit from discoveries that will save their lives. YOU are a hero.